Patients with severe aplastic anemia (SAA) may benefit from hematopoietic stem cell transplantation, but many of them lack a matched donor. Haploidentical transplantation is increasingly utilized for the treatment of nonmalignant disease where patients lack a matched donor. We report patients with aplastic anemia who experienced successful engraftments of haploidentical stem cells with post-transplantation cyclophosphamide (PTCy). Case series and review of the literature. We present two cases of pediatric patients with severe aplastic anemia who experienced successful engraftment of haploidentical related bone marrow. Both patients received conditioning consisting of rabbit ATG, cyclophosphamide, fludarabine, and total body irradiation pretransplant, with PTCy. The conditioning regimen was well tolerated by both patients, and they achieved full donor engraftment and were weaned off all immunosuppressants. Haploidentical stem cell transplantation in patients with severe aplastic anemia may be an effective alternative when fully matched donors are not available. PTCy can facilitate successful engraftment and therefore expand the pool of eligible donors for patients with aplastic anemia.
Keywords: aplastic anemia; haploidentical bone marrow transplant; pediatric; post-transplant cyclophosphamide.
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